Venetoclax 联合低甲基化药物治疗慢性粒单核细胞白血病:倾向得分匹配多中心队列研究

IF 12.8 1区 医学 Q1 HEMATOLOGY Leukemia Pub Date : 2024-11-12 DOI:10.1038/s41375-024-02466-6
Douglas Tremblay, Clifford Csizmar, Courtney D. DiNardo, Somedeb Ball, Noa Rippel, Danielle Hammond, Tapan M. Kadia, Farhad Ravandi, Kelly Chien, Grace Van Hyfte, Madhu Mazumdar, Antoine Saliba, Abhishek Mangaonkar, Terra Lasho, Aref Al-Kali, Marina Kremyanskaya, Jonathan Feld, Lewis R. Silverman, Rami Komrokji, John Mascarenhas, Eric Padron, Guillermo Garcia-Manero, David A. Sallman, Mrinal M. Patnaik, Guillermo Montalban-Bravo
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引用次数: 0

摘要

慢性粒单核细胞白血病(CMML)是一种罕见的血液系统恶性肿瘤,具有骨髓增生异常肿瘤(MDS)和骨髓增生性肿瘤的重叠特征,以外周血单核细胞增多为特征[1]。有向急性髓性白血病(AML)转化的倾向,称为CMML伴胚泡转化(CMML-BT)[2, 3]。低甲基化药物(HMAs)是美国食品及药物管理局(FDA)批准的唯一治疗 CMML 的药物,但在延长总生存期(OS)和阻止疾病演变方面的疗效尚未得到证实 [4,5,6]。为了提高应答率,根据急性髓细胞性白血病(AML)和骨髓增生异常综合征(MDS)的数据,venetoclax(VEN)已与HMAs联合使用[7, 8]。在 CMML 和 CMML-BT 中,单个中心的研究结果不尽相同[9,10,11],但这些研究缺乏对照队列,且受限于在每种治疗环境和疾病类别中接受评估的患者样本量较小。为了明确前期 HMA + VEN 在 CMML 和 CMML-BT 患者中的作用,我们进行了一项多中心回顾性队列研究,利用倾向评分匹配(PSM)队列对单独接受 HMA 治疗的患者进行评估。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Venetoclax in combination with hypomethylating agents in chronic myelomonocytic leukemia: a propensity score matched multicenter cohort study

Chronic myelomonocytic leukemia (CMML) is a rare hematologic malignancy with overlapping features of myelodysplastic neoplasm (MDS) and myeloproliferative neoplasms characterized by peripheral blood monocytosis [1]. There is a predisposition for transformation to acute myeloid leukemia (AML), termed CMML with blast transformation (CMML-BT) [2, 3]. Hypomethylating agents (HMAs) are the sole FDA approved therapy for CMML albeit without established efficacy in terms of prolonging overall survival (OS) and halting disease evolution [4,5,6]. In order to improve response rates, venetoclax (VEN) has been combined with HMAs extrapolating data from AML and MDS [7, 8]. Single centers have offered varied results on the added benefit of VEN to HMA therapy in CMML and CMML-BT [9,10,11], but these studies lack control cohorts and are limited by small sample sizes of patients evaluated in each treatment setting and disease category.

To clarify the role of upfront HMA + VEN in patients with CMML and CMML-BT, we performed a multicenter retrospective cohort study utilizing a propensity score matched (PSM) cohort of patients treated with HMA alone.

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来源期刊
Leukemia
Leukemia 医学-血液学
CiteScore
18.10
自引率
3.50%
发文量
270
审稿时长
3-6 weeks
期刊介绍: Title: Leukemia Journal Overview: Publishes high-quality, peer-reviewed research Covers all aspects of research and treatment of leukemia and allied diseases Includes studies of normal hemopoiesis due to comparative relevance Topics of Interest: Oncogenes Growth factors Stem cells Leukemia genomics Cell cycle Signal transduction Molecular targets for therapy And more Content Types: Original research articles Reviews Letters Correspondence Comments elaborating on significant advances and covering topical issues
期刊最新文献
A CADASIL NOTCH3 mutation leads to clonal hematopoiesis and expansion of Dnmt3a-R878H hematopoietic clones Venetoclax in combination with hypomethylating agents in chronic myelomonocytic leukemia: a propensity score matched multicenter cohort study Endothelial heterogeneity in bone marrow: insights across development, adult life and leukemia Genomic profiling of circulating tumor DNA for childhood cancers The effect of methylation on the let-7-BCL2L1-BCL2 axis and the potential use of hypomethylating and BH3 mimetic drugs in histiocytic neoplasms
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